FDA receives biologics license application for apitegromab in spinal muscular atrophy

A Boston-area biopharmaceutical firm has submitted a biologics license application to the FDA for its investigational, fully human monoclonal antibody to treat individuals with spinal muscular atrophy.

The application includes topline results from the phase 3 SAPPHIRE clinical trial and the phase 2 TOPAZ study examining apitegromab, a muscle-targeted therapy administered to those with spinal muscular atrophy (SMA) already taking a survival motor neuron-targeted treatment, according to a press release from Scholar Rock.

The company has requested priority review from the FDA, which would shorten the application’s review time to 6 months. Apitegromab has previously been granted fast-track, orphan drug and rare pediatric disease designations by the FDA, along with priority medicines and orphan medicinal product designations by the European Medicines Agency.

“We are gratified that in patients already on a [survival motor neuron]-targeted treatment, the SAPPHIRE trial met its primary endpoint for the main efficacy population showing a statistically significant 1.8-point improvement for patients receiving apitegromab compared to placebo,” Jing Marantz, MD, PhD, chief medical officer of Scholar Rock, stated in the release. “With the strength of our phase 3 data as the foundation of our submission, we look forward to continuing to work closely with the FDA.”

Scholar Rock said it expects to investigate apitegromab in patients with SMA aged younger than 2 years in the phase 2 OPAL trial, with a planned initiation by the middle of this year. The trial will evaluate apitegromab in patients who have been or are continuing to be treated with any currently approved therapy such as nusinersen (Spinraza, Biogen) or risdiplam (Evrysdi, Genentech).

The company is also expected to file a marketing authorization application to the EMA by the end of the first quarter.